Y-mAbs Announces Update to National Comprehensive Cancer Network® (NCCN®) Clinical Practice Guidelines in Oncology for Neuroblastoma to Include Naxitamab-gqgk (DANYELZA®)
Y-mAbs Therapeutics (NASDAQ: YMAB) announced that its drug naxitamab-gqgk (DANYELZA®) has been included in the National Comprehensive Cancer Network® (NCCN®) Clinical Practice Guidelines as a Category 2A treatment option for high-risk neuroblastoma. The drug, which received FDA accelerated approval in November 2020, is used in combination with GM-CSF for treating relapsed or refractory high-risk neuroblastoma in pediatric patients (1+ years) and adults.
DANYELZA's FDA approval was based on efficacy results from two single-arm, open-label trials: Study 201 and Study 12-230. The drug was developed by Memorial Sloan Kettering Cancer Center and is exclusively licensed to Y-mAbs. This NCCN recognition reinforces DANYELZA's position as a leading anti-GD2 therapy for treating relapsed/refractory high-risk neuroblastoma.
Y-mAbs Therapeutics (NASDAQ: YMAB) ha annunciato che il suo farmaco naxitamab-gqgk (DANYELZA®) è stato incluso nelle Linee Guida Cliniche della National Comprehensive Cancer Network® (NCCN®) come opzione terapeutica di Categoria 2A per il neuroblastoma ad alto rischio. Il farmaco, che ha ricevuto l'approvazione accelerata dalla FDA nel novembre 2020, viene utilizzato in combinazione con GM-CSF per il trattamento del neuroblastoma ad alto rischio recidivante o refrattario in pazienti pediatrici (da 1 anno in su) e adulti.
L'approvazione della FDA per DANYELZA si basa sui risultati di efficacia di due studi a braccio singolo e in aperto: Studio 201 e Studio 12-230. Il farmaco è stato sviluppato dal Memorial Sloan Kettering Cancer Center ed è concesso in licenza esclusiva a Y-mAbs. Questo riconoscimento da parte della NCCN rafforza la posizione di DANYELZA come terapia anti-GD2 di riferimento per il trattamento del neuroblastoma ad alto rischio recidivante/refrattario.
Y-mAbs Therapeutics (NASDAQ: YMAB) anunció que su medicamento naxitamab-gqgk (DANYELZA®) ha sido incluido en las Guías de Práctica Clínica de la National Comprehensive Cancer Network® (NCCN®) como una opción de tratamiento de Categoría 2A para el neuroblastoma de alto riesgo. El medicamento, que recibió la aprobación acelerada de la FDA en noviembre de 2020, se utiliza en combinación con GM-CSF para tratar el neuroblastoma de alto riesgo recidivante o refractario en pacientes pediátricos (mayores de 1 año) y adultos.
La aprobación de la FDA para DANYELZA se basó en los resultados de eficacia de dos ensayos abiertos de brazo único: Estudio 201 y Estudio 12-230. El medicamento fue desarrollado por el Memorial Sloan Kettering Cancer Center y está licenciado exclusivamente a Y-mAbs. Este reconocimiento de la NCCN refuerza la posición de DANYELZA como una terapia líder anti-GD2 para el tratamiento del neuroblastoma de alto riesgo recidivante/refractario.
Y-mAbs Therapeutics (NASDAQ: YMAB)는 자사의 약물 naxitamab-gqgk (DANYELZA®)가 National Comprehensive Cancer Network®(NCCN®) 임상 진료 지침에 고위험 신경모세포종 치료를 위한 2A 등급 치료 옵션으로 포함되었다고 발표했습니다. 이 약물은 2020년 11월 FDA의 가속 승인(Accelerated Approval)을 받았으며, GM-CSF와 병용하여 소아(1세 이상) 및 성인의 재발 또는 불응성 고위험 신경모세포종 치료에 사용됩니다.
DANYELZA의 FDA 승인은 단일군, 공개 라벨 임상시험인 연구 201 및 연구 12-230의 효능 결과를 기반으로 합니다. 이 약물은 Memorial Sloan Kettering Cancer Center에서 개발되었으며 Y-mAbs에 독점 라이선스가 부여되었습니다. 이번 NCCN 인정은 재발/불응성 고위험 신경모세포종 치료를 위한 선도적인 항-GD2 치료제로서 DANYELZA의 입지를 강화합니다.
Y-mAbs Therapeutics (NASDAQ : YMAB) a annoncé que son médicament naxitamab-gqgk (DANYELZA®) a été inclus dans les recommandations cliniques de la National Comprehensive Cancer Network® (NCCN®) en tant qu'option de traitement de catégorie 2A pour le neuroblastome à haut risque. Ce médicament, qui a reçu une approbation accélérée de la FDA en novembre 2020, est utilisé en association avec le GM-CSF pour traiter le neuroblastome à haut risque récurrent ou réfractaire chez les patients pédiatriques (à partir de 1 an) et les adultes.
L'approbation de la FDA pour DANYELZA s'appuie sur les résultats d'efficacité de deux essais ouverts à un seul bras : l'étude 201 et l'étude 12-230. Ce médicament a été développé par le Memorial Sloan Kettering Cancer Center et est sous licence exclusive à Y-mAbs. Cette reconnaissance par la NCCN renforce la position de DANYELZA en tant que thérapie anti-GD2 de référence pour le traitement du neuroblastome à haut risque récurrent/réfractaire.
Y-mAbs Therapeutics (NASDAQ: YMAB) gab bekannt, dass sein Medikament naxitamab-gqgk (DANYELZA®) in die Clinical Practice Guidelines des National Comprehensive Cancer Network® (NCCN®) als Behandlungsmöglichkeit der Kategorie 2A für Hochrisiko-Neuroblastom aufgenommen wurde. Das Medikament, das im November 2020 eine beschleunigte Zulassung der FDA erhielt, wird in Kombination mit GM-CSF zur Behandlung von rezidiviertem oder refraktärem Hochrisiko-Neuroblastom bei pädiatrischen Patienten (ab 1 Jahr) und Erwachsenen eingesetzt.
Die FDA-Zulassung von DANYELZA basierte auf Wirksamkeitsergebnissen aus zwei einarmigen, offenen Studien: Studie 201 und Studie 12-230. Das Medikament wurde vom Memorial Sloan Kettering Cancer Center entwickelt und ist exklusiv an Y-mAbs lizenziert. Diese Anerkennung durch die NCCN stärkt die Position von DANYELZA als führende Anti-GD2-Therapie zur Behandlung von rezidivierendem/refraktärem Hochrisiko-Neuroblastom.
- DANYELZA included in NCCN Guidelines as Category 2A treatment option for high-risk neuroblastoma
- Recognition by NCCN strengthens DANYELZA's position as a leading anti-GD2 therapy
- Drug has FDA accelerated approval for both pediatric and adult patients
- None.
Insights
NCCN guideline inclusion of DANYELZA for high-risk neuroblastoma represents significant clinical validation, potentially expanding treatment options for this rare pediatric cancer.
The inclusion of naxitamab-gqgk (DANYELZA) in the NCCN Guidelines as a Category 2A treatment option marks a significant clinical milestone for patients with high-risk neuroblastoma. This designation indicates there is NCCN consensus that this intervention is appropriate based on available evidence. For context, neuroblastoma is particularly challenging in the relapsed or refractory setting, where effective treatment options are limited.
DANYELZA targets GD2, which is expressed on neuroblastoma cells, positioning it as what the company describes as "a leading anti-GD2 therapy of choice" for physicians. The drug received FDA accelerated approval in November 2020 specifically for pediatric patients (age 1+) and adults with relapsed/refractory high-risk neuroblastoma in the bone or bone marrow who demonstrated partial response, minor response, or stable disease to prior therapy.
The NCCN Guidelines are widely respected and referenced by oncologists when making treatment decisions. This inclusion may help standardize the use of DANYELZA in appropriate patient populations, providing physicians with additional confidence when considering treatment options for this challenging disease. The approval pathway and subsequent guideline inclusion reflect the ongoing efforts to address the significant unmet needs that still exist in pediatric oncology, particularly for aggressive and difficult-to-treat malignancies like high-risk neuroblastoma.
From a commercial perspective, the inclusion of DANYELZA in the NCCN Guidelines represents a positive development for Y-mAbs Therapeutics. NCCN guideline inclusion is a significant milestone that typically enhances a therapy's clinical adoption potential, as these guidelines directly influence treatment protocols at major cancer centers across the US.
For Y-mAbs, a company focused on developing novel cancer therapies, having their product recognized with Category 2A status provides important external validation. This designation comes approximately 4.5 years after DANYELZA's accelerated FDA approval in November 2020, suggesting the therapy has gained sufficient clinical experience and recognition to warrant inclusion in these influential guidelines.
While the press release doesn't disclose commercial figures, NCCN guideline inclusion generally supports reimbursement discussions with payers, as many insurance companies consider these guidelines when determining coverage policies. For rare disease treatments like those for high-risk neuroblastoma, guideline inclusion can be particularly valuable in establishing a therapy as a standard treatment option.
This development strengthens DANYELZA's position within Y-mAbs' commercial portfolio and supports the company's focus on radioimmunotherapy and antibody-based therapeutic products. For investors following Y-mAbs, this represents a positive clinical and commercial advancement that enhances the potential utilization of one of the company's key approved products.
PRINCETON, N.J., May 07, 2025 (GLOBE NEWSWIRE) -- Y-mAbs Therapeutics, Inc. (the “Company” or “Y-mAbs”) (Nasdaq: YMAB), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel radioimmunotherapy and antibody-based therapeutic products for the treatment of cancer, today announced that naxitamab-gqgk (DANYELZA®) is recommended by the National Comprehensive Cancer Network® (“NCCN”) Clinical Practice Guidelines in Oncology (NCCN Guidelines®) as a NCCN Category 2A treatment option for high-risk neuroblastoma.
“We are very pleased with NCCN’s update of the NCCN Guidelines to include naxitamab-gggk (DANYELZA). We believe this decision reinforces the importance of naxitimab-gggk (DANYELZA) as a leading anti-GD2 therapy of choice for physicians treating patients with relapsed/refractory high-risk neuroblastoma,” said Doug Gentilcore, SVP, DANYELZA Business Unit Head.
Naxitamab-gqgk (DANYELZA) was granted accelerated approval by the U.S. Food and Drug Administration (“FDA”) on November 25, 2020 in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) for pediatric patients one year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow demonstrating a partial response, minor response, or stable disease to prior therapy. The FDA approval of naxitamab-gqgk (DANYELZA) was based on efficacy results in patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow enrolled in two single-arm, open-label trials: Study 201 (NCT 03363373) and Study 12-230 (NCT 01757626).
The NCCN is a not-for-profit alliance of 33 leading cancer centers devoted to patient care, research, and education. NCCN is dedicated to defining and advancing quality, effective, equitable, and accessible cancer care and prevention so all people can live better lives. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.
Researchers at Memorial Sloan Kettering Cancer Center (“MSK”) developed naxitamab-gqgk (DANYELZA), which is exclusively licensed by MSK to Y-mAbs. MSK has institutional financial interests related to the compound and Y-mAbs.
About DANYELZA® (naxitamab-gqgk)
DANYELZA® (naxitamab-gqgk) is indicated, in combination with granulocyte-macrophage colony-stimulating factor ("GM-CSF"), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. This indication was approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefits in a confirmatory trial. DANYELZA® includes a Boxed Warning for serious infusion-related reactions, such as cardiac arrest and anaphylaxis, and neurotoxicity, such as severe neuropathic pain and transverse myelitis. See full Prescribing Information for complete Boxed Warning and other important safety information here.
DANYELZA is currently not approved for the treatment of osteosarcoma in any jurisdiction.
About Y-mAbs
Y-mAbs is a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, radioimmunotherapy and antibody-based therapeutic cancer products. The Company’s technologies include its investigational Self-Assembly DisAssembly (“SADA”) Pretargeted Radioimmunotherapy Platform (“PRIT”) and bispecific antibodies generated using the Y-BiClone platform. The Company’s broad and advanced product pipeline includes the anti-GD2 therapy DANYELZA® (naxitamab-gqgk), the first FDA-approved treatment for patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow after a partial response, minor response, or stable disease to prior therapy.
Forward-Looking Statements
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements include, but are not limited to, statements about our business model, including financial outlook for 2025 and beyond, including estimated operating expenses, use of cash and cash equivalents and DANYELZA product revenue and sufficiency of cash resources and related assumptions; expectations with respect to the Company’s future financial performance; implied and express statements regarding the future of the Company’s business, including with respect to expansion and its goals; expectations with respect to the Company’s plans and strategies, development, regulatory, commercialization and product distribution plans, including the timing thereof; expectations with respect to the Company’s products and product candidates, including potential territory and label expansion of DANYELZA and the potential market opportunity related thereto and potential benefits thereof, and the potential of the SADA PRIT technology and potential benefits and applications thereof; expectations relating to key anticipated development milestones, including potential expansion and advancement of commercialization and development efforts, including potential indications, applications and geographies, and the timing thereof; expectations with respect to current and future clinical and pre-clinical studies and the Company’s research and development programs, including with respect to timing and results; expectations regarding collaborations or strategic partnerships and the potential benefits thereof; and other statements that are not historical facts. Words such as ‘‘anticipate,’’ ‘‘believe,’’ “contemplate,” ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ “hope,” ‘‘intend,’’ ‘‘may,’’ ‘‘might,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘should,’’ ‘‘target,’’ “will,” ‘‘would’,’ “guidance,” “goal,” “objective,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Our product candidates and related technologies are novel approaches to cancer treatment that present significant challenges. Actual results may differ materially from those indicated by such forward-looking statements as a result of various factors, including but not limited to: risks associated with the Company’s financial condition and need for additional capital; the risks that actual results of the Company’s restructuring plan and revised business plan will not be as expected; risks associated with the Company’s development work; cost and success of the Company’s product development activities and clinical trials; the risks of delay in the timing of the Company’s or its partners’ regulatory submissions or failure to receive approval of its drug candidates; the risks related to commercializing any approved pharmaceutical product including the rate and degree of market acceptance of product candidates; development of sales and marketing capabilities and risks associated with failure to obtain sufficient reimbursement for products; risks related to the Company’s dependence on third parties including for conduct of clinical testing and product manufacture as well as regulatory submissions; the Company’s ability to enter into new partnerships or to recognize the anticipated benefits from its existing partnerships; risks related to government regulation; risks related to market approval, risks associated with protection of the Company’s intellectual property rights; risks related to employee matters and managing growth; risks related to the Company’s common stock, risks associated with macroeconomic conditions, including the conflict between Russia and Ukraine and sanctions related thereto, the state of war between Israel and Hamas and the related risk of a larger regional conflict, inflation, increased interest rates, uncertain global credit and capital markets and disruptions in banking systems; and other risks and uncertainties affecting the Company including those described in the “Risk Factors” section included in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2024, and the Company’s Quarterly Report on Form 10-Q for the quarterly periods ended March 31, 2024, and September 30, 2024, and future filings and reports by the Company. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company undertakes no obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
SADA®, SADA PRIT™, DANYELZA® and Y-mAbs® are registered trademarks of Y-mAbs Therapeutics, Inc.
Investor Contact: Courtney Dugan VP, Head of Investor Relations cdu@ymabs.com
FAQ
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